BCX9930 in previously C5 treated PNH patients

• Research type

  Research Study

• Full title

  A Randomized, Open-Label, Multicenter, Parallel-Group Study to Evaluate the Efficacy, Safety, and Tolerability of Oral BCX9930 Monotherapy for the Treatment of Paroxysmal Nocturnal Hemoglobinuria in Subjects with Inadequate Response to C5 Inhibitor Therapy

• IRAS ID

  298286

• Contact name

  Mary Katherine Farmer

• Contact email

  mm.pnh@biocryst.com

• Sponsor organisation

  BioCryst Pharmaceuticals Inc

• Eudract number

  2020-004438-39

• Clinicaltrials.gov Identifier

  NCT05116774

• Duration of Study in the UK

  3 years, 3 months, 29 days

• Research summary

  The clinical trial BCX9930-202 will investigate if, in PNH patients, BCX9930 oral therapy is better than C5 inhibitor treatment (given via iv infusions), over a 24 week period, followed by a 28 week treatment period with all patients on BCX9930 to assess long term safety and tolerability.
  PNH patients who are receiving the standard of care, a C5 inhibitor, can still experience uncontrolled disease, with low haemoglobin levels and often a continued need for transfusions.
  This study will assess a new oral treatment BCX9930 with a different mode of action to the current standard of care (C5 inhibitors).
  Enrolled patients will have been treated with C5 inhibitors, but remain in need of further therapy. The study treatment will last 52 weeks and involve at least monthly visits to the research sites for blood tests and physical examinations

• REC name

  North East - Tyne & Wear South Research Ethics Committee

• REC reference

  21/NE/0119

• Date of REC Opinion

  5 Jul 2021

• REC opinion

  Favourable Opinion